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FDA Grants Breakthrough Therapy Designation to Mirum's Volixibat for Cholestatic Pruritus in PBC

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  Mirum Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough therapy designation to their drug, volixibat, as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). This designation was granted following positive interim results from the Phase 2b VANTAGE study, which demonstrated statistically significant improvements in pruritus symptoms compared to a placebo for patients treated with volixibat. Publish Date: 10-10-2024   Source: Mirum Pharmaceuticals, Inc. Primary biliary cholangitis (PBC), once known as primary biliary cirrhosis, is a chronic ailment involving progressive damage, inflammation, and eventual destruction of the liver’s small bile ducts. Bile, a crucial digestive fluid produced by the liver, plays a vital role in processing food and expelling toxins, cholesterol, and aged red blood cells from the body. However, when these bile ducts suffer harm, bile accu...

Primary Biliary Cholangitis (PBC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

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 Primary biliary cholangitis (PBC), once known as primary biliary cirrhosis, is a chronic ailment involving progressive damage, inflammation, and eventual destruction of the liver's small bile ducts. Bile, a crucial digestive fluid produced by the liver, plays a vital role in processing food and expelling toxins, cholesterol, and aged red blood cells from the body. However, when these bile ducts suffer harm, bile accumulates in the liver, leading to irreversible scarring of liver tissue, a condition known as cirrhosis. Activated CD4 and CD8 lymphocytes drive the continual deterioration of these small and medium-sized bile ducts. In adults experiencing cholestasis (elevated levels of alkaline phosphatase (ALP) or gamma-glutamyl transferase (GGT)), suspicions of PBC arise once obstructive jaundice and systemic diseases have been ruled out through abdominal ultrasound. A confirmed diagnosis is made when disease-specific autoantibodies, such as antimitochondrial autoantibodies (AMA tit...

Primary Biliary Cholangitis (PBC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

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  Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is a chronic condition in which the liver's tiny bile ducts become damaged, inflamed, and eventually destroyed. Bile is a fluid made in the liver. It aids digestion and helps the body eliminate toxins, cholesterol and worn-out red blood cells. When the bile ducts are harmed, bile can back up in the liver, causing irreversible scarring of the liver tissue (cirrhosis). The continuous destruction of small and medium bile ducts is mediated by activated CD4 and CD8 lymphocytes. The prevalence of PBC has been estimated to be approximately 51 to 65 cases per 100,000 population in the United States. Thelansis’s “Primary Biliary Cholangitis (PBC) Market Outlook, Epidemiology, Competitive Landscape , and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, ...